Hope Of Patients With Rare Diseases Under "No Medicine" And "High Price Medicine": Multi Payment Assistance Program

"No medicine" and "can't afford" have become the biggest obstacle for patients with rare diseases to seek medical treatment. This is a special medicine for spinal cord atrophy.

In this regard, at the second rare disease cooperation and exchange meeting recently, Zhang Wenbao, director of the medical administration bureau of the National Health Commission, pointed out that the prevention and control of rare diseases involves the construction of hierarchical diagnosis and treatment system, the construction of drug supply security system, and the reform of medical insurance payment mode. The steady promotion of rare disease prevention and control involves a wide range of aspects, and requires the full cooperation of multiple government departments and social forces.

Wu Xiaobin, general manager and President of Baiji Shenzhou China region, pointed out to the 21st century economic reporter that the most important thing in medicine is policy. As long as the policy is right, there will be no problem in the development of rare disease medicine in China, such as simplifying the examination and approval, simplifying the process, and medical insurance can quickly keep up with it. Professor Shi Luwen, director of the International Research Center for medical management of Peking University, pointed out that in terms of medical security, the mode of government guidance, multi-party participation and third-party win-win situation, or the system that China must find ways to build in the future.

Wang yiou, Secretary General of the Beijing pain challenge foundation, also pointed out to the 21st century economic reporter that the challenges faced by patients with rare diseases involve many aspects, which are extremely complex and can not be solved by any party alone. "The research, diagnosis and treatment of rare diseases, payment and guarantee of treatment expenses for rare diseases, research and development, clinical and admittance of related drugs, genetic inheritance and fertility of rare diseases, social support systems such as psychology, education and employment of patients, and cognition of rare diseases in the whole society are all common problems faced by rare disease groups."

It is difficult to use drugs in patients with rare diseases

"The vast majority of patients with rare diseases have been misdiagnosed on their way to medical treatment, because the disease is too few, so the doctors do not know, and it is difficult to make a diagnosis. 99% of them have no medicine; 1% of them have medicine, which is foreign medicine, but not in China, so they can't get in. It's not easy for us to come in and it's too expensive to buy without medical insurance." These are not easy to describe the difficult road of seeking medical treatment for patients with rare diseases.

If the special effect of mucopolysaccharide was introduced into the market in China in 2020, she would have told her that polysaccharide would have been used for the diagnosis and treatment of mucopolysaccharide type I in the 21st century.

"I've been waiting for this medicine for 17 years, and there's no medicine during this period. Now I've got skeletal deformity, corneal opacity and other symptoms. On June 3, the State Drug Administration approved the listing of aierzan through the priority review and approval process. I hope the price can be more friendly in the future. " Zhang Xiao pointed out.

Not long ago, Wu Shiping, a 32 year old citizen in Huaihua City, Hunan Province, also asked for help on the Internet. His son, who is more than one year old, suffers from spinal muscular atrophy and has been lying in the ICU of the hospital for several months. He is in urgent need of an injection of a specific drug called nosinasheng sodium, which costs about 700000 yuan per injection.

In fact, high drug price has become a big problem for patients.

According to statistics, about 7000 rare diseases have been confirmed worldwide. According to the definition given by the genetics branch of the Chinese Medical Association, rare diseases are diseases or diseases with a prevalence of less than one in 500000 or a neonatal incidence of less than one in ten thousand. According to this estimate, there are more than 20 million patients with rare diseases in China. Many of them are from childhood, and their conditions are often serious.

According to the China rare disease drug accessibility report (2019), 42% of the patients did not receive any treatment from 2014 to 2018, while the vast majority of the patients who received treatment failed to take the treatment drugs timely and adequately; more than half of the patients were disabled due to illness, of which 29% were physically disabled, 15% were multiple disabilities (i.e., there were more than two types of disability at the same time); 80% of the patients were disabled due to diseases The annual family income of patients is less than 50000 yuan, and more than half of the patients spend 80% of the annual family income on disease treatment, which is far beyond the 40% safety threshold defined by the World Health Organization.

Due to the high price of rare diseases, Du Xiangyang, chief analyst of Southwest Securities pharmaceutical, said that the number of patients with a single rare disease is small, so the number of patients suitable for the drug is small, and the market demand for a single disease is low. However, at present, the research and development costs of innovative drugs are high, so it is difficult for enterprises to recover the R & D costs, which has hit the enthusiasm of enterprises in R & D to a certain extent.

In addition to economic pressure, patients with rare diseases also face mental pressure. For example, a person in charge of aixike, a public welfare organization for pulmonary hypertension, told the reporter of the 21st century economic report: "patients with pulmonary hypertension are a kind of pulmonary vascular disease with a high degree of malignancy and can not be cured at present. Dyspnea, shortness of breath, suffocation and hemoptysis are common symptoms. With the continuous increase of pulmonary vascular resistance in patients, heart and respiratory failure can eventually be caused These patients usually do not have particularly significant symptoms. Except for the blue lips, the symptoms are "recessive". They are often misunderstood as lazy when they are working, and they are also rejected when applying for the minimum living allowance

Multi party co payment scheme launched

According to Wang yiou, the challenges faced by patients with rare diseases include research, diagnosis and treatment of rare diseases, payment and guarantee of treatment expenses for rare diseases, research and development, clinical and admission of related drugs, genetic inheritance and fertility of rare diseases, social support systems such as psychology, education and employment of patients, and cognition of rare diseases in the whole society All these problems need to be solved together.

Zhang Wenbao pointed out that in view of rare diseases, one is to focus on prevention, and most of them are congenital and genetic diseases. Through premarital examination, prenatal examination and neonatal disease screening, we can reduce the occurrence of birth defects and reduce the long-term health damage caused by diseases to newborns; the second is to implement classified measures. The third is to steadily promote the prevention and treatment of rare diseases, involving a wide range of government departments and social forces to work together.

At present, in order to solve the core problem of drug use and payment, the government, pharmaceutical enterprises and charitable organizations are also making joint efforts.

In order to help patients with rare diseases solve their medication problems, for example, relevant departments have successively issued regulations and implementation measures in respect of VAT reduction, special examination and approval, special channels, priority review, and acceleration of listing, which greatly accelerated the speed of foreign rare disease drugs entering China and benefiting Chinese patients.

In terms of payment, national health care administrators are also trying to include high-priced drugs in the health insurance list through negotiation. In 2017, the Ministry of human resources and social security for the first time included two orphan drugs (recombinant human coagulation factor VIIa for hemophilia and recombinant human interferon beta 1b for multiple sclerosis) into the medical insurance list through the national negotiation mechanism. In 2018, the State Council's institutional reform established the National Medical Security Bureau, which is responsible for the management of medical insurance. In 2019, the national medical insurance catalogue operated by the National Health Insurance Bureau was updated, and rare disease drugs were included for the first time.

However, the payment of rare diseases can not only rely on national health insurance.

On July 31, the State Medical Insurance Bureau deliberated and promulgated the "Interim Measures for the administration of drug use in basic medical insurance". Article 4 of the Interim Measures points out that we should adhere to the functional orientation of "basic medical insurance", do our best and do what we can, and the level of medication security should be compatible with the basic medical insurance fund and the affordability of the insured.

Professor Shi Luwen pointed out that in terms of medical security, the mode of government guidance, multi-party participation and third-party win-win, or the system that China must find ways to build in the future.

Wang yiou put forward the concept of "value co creation", that is, patients, doctors, government, enterprises and other parties can explore different modes, and there are different leading parties in different modes. We should promote all relevant parties to participate in and work together to find the best way to overcome rare diseases or coexist peacefully with rare diseases, seek the maximization of patients' health, and finally share the benefits What we have achieved.

"As a charity, we can make suggestions from the perspective of social philanthropy, together with the support of enterprises, commercial insurance and medical insurance policies, so as to reduce the burden of patients more effectively." Wang further explained that this multi-party co payment model has been recognized by the local government.

"In accordance with this idea, we launched two local special pilot projects in Zhejiang and Shanxi in the first half of this year, and have started to receive assistance applications from local patients. Through the multi-party co payment model, the pressure of the government is reduced, and the confidence of patients' families is improved. In the second half of the year, we will continue to promote the medical assistance project for rare diseases to more regions, and such a project is a typical development resource model. That is to say, patients' organizations should take the lead and participate in creating new methods conducive to problem-solving, which can promote the whole problem to be better solved. " Wang yiou said.

At the same time, Wu Xiaobin pointed out to the 21st century economic reporter that if there is national policy support, enterprises are also willing to participate. "Every country has a special policy for rare diseases, because the number of rare patients is very small, and the research and development cost, time and investment are very large. If there are special policies, such as simplifying the approval and simplifying the process, the medical insurance can quickly keep up with it, and we will have the motivation to research and develop again. It is hoped that the policy can be improved as soon as possible to better support the research and development of drugs for rare diseases in China. "

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